Benitec Biopharma Inc. develops clinical-stage genetic medicines based on its proprietary Silence and Replace DNA-directed RNA interference platform. The company’s lead program, BB-301, is a silence-and-replace therapeutic in development for dysphagia in patients with Oculopharyngeal Muscular Dystrophy, or OPMD.
Company news primarily covers BB-301 clinical development, including Phase 1b/2a study updates, follow-up data from treated cohorts, safety and clinical-activity observations, and presentations at gene therapy and muscular dystrophy medical meetings. Benitec also issues periodic financial results and operational updates tied to its development-stage biotechnology business.
Benitec Biopharma Inc. (NASDAQ: BNTC) has released its full year 2024 financial results and operational update. Key highlights include:
- Positive 90-day and 180-day interim clinical trial data for the first OPMD subject dosed with low-dose BB-301
- Second subject dosed in February 2024, third expected in Q4 2024
- Closed $40.0 million private placement financing in April, extending cash runway through 2025
- Total revenues were $0 for FY2024, compared to $75,000 in FY2023
- Total expenses increased to $22.5 million in FY2024 from $19.2 million in FY2023
- Net loss of $22.4 million, or $5.51 per share, compared to $19.6 million, or $14.12 per share in FY2023
- Cash and cash equivalents of $50.9 million as of June 30, 2024
Benitec Biopharma Inc. (NASDAQ: BNTC) announced that a Late Breaking Abstract on BB-301 has been accepted for oral presentation at the 29th Annual Congress of the World Muscle Society in Prague, Czech Republic, from October 8-12, 2024. The presentation, scheduled for October 12, 2024, will focus on interim clinical data from a Phase 1b/2a study evaluating the safety and clinical activity of BB-301, an AAV9-based gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia. Professor Milan R. Amin, M.D., from the New York University Grossman School of Medicine, will present the findings. This presentation represents a significant milestone in Benitec's development of novel genetic medicines using their proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform.
Benitec Biopharma (NASDAQ: BNTC) announced that CEO Jerel A. Banks, M.D., Ph.D., will participate in the Oculopharyngeal Muscular Dystrophy (OPMD) Awareness Day Webinar on September 23, 2024, at 12 PM EST. The event, organized by the OPMD Association, aims to raise awareness about OPMD, a rare, genetic, adult-onset, progressive myopathy.
Dr. Banks will provide an update on the BB-301 Phase 1b/2a clinical development program. The webinar will offer patients, families, and caregivers clinical insights, patient perspectives, and informational resources to support understanding and care for OPMD. Benitec Biopharma is a clinical-stage biotechnology company developing novel genetic medicines based on its proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform.
Benitec Biopharma (NASDAQ: BNTC) reports continued improvements in swallowing efficiency for the first OPMD subject treated with low-dose BB-301 in their Phase 1b/2a study. At the 180-day post-dose assessment, Total Pharyngeal Residue (TPR) remained meaningfully reduced compared to pre-dose values. For three of four food types, TPR values were lower than at any point during the 9-month pre-dose observation period.
The Sydney Swallow Questionnaire (SSQ) Total Score showed continued reductions in dysphagic symptoms, indicating improved swallowing function. Both TPR and SSQ scores are considered central markers for evaluating clinical improvement in OPMD patients. The company plans to dose the third subject in Q3 2024 and report additional interim results in Q1 2025.
Benitec Biopharma, a clinical-stage gene therapy-focused biotechnology company, has appointed Kishen Mehta to its board of directors, effective June 26, 2024. This follows a $40 million PIPE financing led by Suvretta Capital, where Mehta is a portfolio manager. CEO Jerel A. Banks emphasized Mehta's expertise in investing, corporate development, and strategy, particularly as the company advances its Phase 1b/2a study for BB-301, a treatment for Oculopharyngeal Muscular Dystrophy (OPMD). Mehta brings over 15 years of financial industry experience, including roles at Biohaven Pharmaceuticals, Surveyor Capital, and Adage Capital, with a focus on healthcare investments. He expressed enthusiasm for contributing to Benitec's mission to develop effective treatments for OPMD patients.
Benitec Biopharma reported positive interim clinical trial data for the first Oculopharyngeal Muscular Dystrophy (OPMD) subject dosed with BB-301, with improvements noted in swallowing assessments. The company closed an oversubscribed $40.0 million private financing round extending cash runway through 2025. Operational updates include enrollment of 23 subjects in the OPMD Natural History Study and milestones related to BB-301 development.
Benitec Biopharma Inc. (BNTC) will participate in the Citizens JMP Life Science Conference in New York on May 13-14, 2024, presenting its gene therapy-focused ddRNAi platform. The presentation by Jerel Banks, MD, PhD, Executive Chairman and CEO, will be webcasted live.
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